A plea for availability, accessibility and affordability of new therapies

You may have attended or heard about the tremendous advances which were reported during the last ASH 2017 meeting held in Atlanta. The hematology field is moving very rapidly compared to other medical disciplines, and we have never been in a better position to advance treatments and reach cure in several life-threatening diseases.

However, our excitement and enthusiasm about these amazing innovative therapies should not obscure the difficulties and challenges that patients and health care professionals are increasingly facing. It is about the ‘triple-A dilemma’ related to “Availability”, “Accessibility” and “Affordability” of novel therapies to all patients all around the globe.

“Availability”: more and more, we see a recurrent sudden shortage of many essential drugs which have been routinely used for several decades as standard of care. Most of the time, little information is provided about the exact reasons for such shortage, which is seriously impacting patients. Many of these “old” drugs will “re-appear” here and there, but at a relatively higher cost.

“Accessibility”: therapeutic innovations should reach all patients who are in need in a timely manner. Most of these life-threatening diseases require urgent intervention. Lengthy procedures should be simplified and priority should be put on the approval of trials aiming to make a true and genuine difference for patients. Extending overall survival with few side effects and good quality of life should be the ultimate goals of any treatment

“Affordability”: the announced price tags for most novel drugs and innovative cellular therapies appear to be quite prohibitive, not taking into account the cost of management of potentially severe complications, which may add another substantial expenditure on top of that of the innovative product itself. Reimbursement of such exorbitant costs is unlikely to happen in most developed or rich countries, not to mention those with low- to middle-income. Such cost concerns will deepen inequality between patients, and lead to impossibility of or unacceptably delayed access for the majority.

We, undersigned, health care professionals from the EBMT and other medical and scientific organizations, ask urgently all stake holders to:

(i) recognize the magnitude and complexity of the above problems which are already harming our patients;

(ii) recognize the vital need and challenges related to the development of innovative therapies for life-threatening diseases;

(iii) avoid simplistic solutions and “who to blame” statements, but rather tackle the challenges through a sincere, deep and constructive dialogue between manufacturers, regulators, payers, health care professionals and patients’ representatives in order to preserve patients’ interest at all time;

(iv) identify temporary organizational and technical solutions to allow for “Availability”, “Accessibility” and “Affordability” of innovative therapies, while waiting for long-term solutions in a progressively transforming health system intent on solving the ‘triple-A dilemma’.

We are fully committed to defending patients’ interests at all time, and we will act in accordance because health care for life-threatening diseases should not be transformed into an exclusive profit-driven marketplace.

Mohamad Mohty

EBMT President

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Medical and scientific organizations: A plea for availability, accessibility and affordability of new therapies